RA Capital Management

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Trace Neuroscience is a biopharmaceutical company dedicated to advancing genetic medicine for patients with neurodegenerative diseases. It specializes in developing targeted antisense oligonucleotide therapies, with its lead product aimed at restoring UNC13A protein function in individuals with Amyotrophic Lateral Sclerosis (ALS) caused by TDP-43 mislocalization. This therapy corrects UNC13A mRNA splicing, potentially improving muscle function and extending survival for these patients.

Gameto is a biotechnology company focused on improving female reproductive health through innovative therapies. Recognizing that women's health is an underserved area with limited treatment options, Gameto aims to develop advanced solutions that enhance patient outcomes. The company utilizes cellular engineering to create female reproductive cell lines, employing proprietary combinations of transcription factors to reprogram human induced pluripotent stem cells (hiPSCs) into engineered ovarian and endometrial cell lines that function similarly to natural cells. This platform supports a portfolio of cellular therapeutics targeting various female reproductive diseases. Gameto's flagship program, Fertilo, is designed to enhance the processes of in vitro fertilization (IVF) and egg freezing, making them shorter, safer, and more effective. Additional applications of Gameto's technology include Deovo, an organoid of the female reproductive system, and Ameno, a cell-based therapeutic aimed at alleviating the health issues associated with primary ovarian insufficiency and menopause.

Ajax Therapeutics is a biotechnology company based in New York City that focuses on developing innovative small molecule therapies for hematologic malignancies. The company utilizes computational chemistry and structure-based technologies to create targeted treatments that address critical cytokine signaling pathways involved in these diseases. By integrating insights from disease mechanisms, genetics, and structural biology with an advanced computational drug discovery platform, Ajax Therapeutics aims to design precise therapeutics that meet significant unmet medical needs for patients suffering from hematologic malignancies.

Lexeo Therapeutics is a biotechnology company specializing in adeno-associated virus (AAV)-mediated therapies for both rare and common diseases caused by single gene mutations. Headquartered in New York, the company maintains an integrated pipeline developed in collaboration with Weill Cornell Medicine's Department of Genetic Medicine. Lexeo focuses on advancing its clinical programs towards commercialization while continuing to research new therapies for various patient populations and unmet medical needs.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

Avidity Biosciences, Inc. is a biotechnology company focused on developing oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These innovative therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. This approach allows Avidity to target previously undruggable tissue and cell types, thereby addressing the underlying genetic drivers of various diseases. The company's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, a rare muscle disease. Additionally, Avidity is advancing multiple programs aimed at treating muscle-related conditions such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Founded in 2012 and based in La Jolla, California, Avidity is also exploring therapeutic applications in immune and other cell types.

BioAge Labs, Inc. is a biotechnology company focused on developing therapies to address aging and age-related diseases. The company utilizes a systems biology and artificial intelligence platform to identify key drug targets that influence aging and analyze extensive datasets to uncover the molecular drivers of age-related conditions. BioAge Labs is advancing several drug candidates, including BGE-117, a hypoxia-inducible factor prolyl hydroxylase inhibitor designed to activate genes involved in critical biological processes such as tissue regeneration and vascular remodeling. Additionally, BGE-175, an orally administered inhibitor of the prostaglandin D2 DP1 signaling pathway, aims to mitigate risks associated with immune aging and respiratory infections. Founded in 2015 and based in Richmond, California, BioAge Labs is committed to discovering and developing innovative treatments that harness the biology of human aging.

PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing nucleic acid therapeutics, particularly antisense oligonucleotides. Founded in 2018, the company aims to enhance the delivery and efficacy of these therapies through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative platform utilizes engineered peptides designed to improve tissue penetration, cellular uptake, and nuclear delivery of oligonucleotides. PepGen's technology has shown promise in preclinical studies, demonstrating the ability to transport therapeutics effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. The company's mission is to unlock the full potential of nucleic acid therapeutics to transform the treatment landscape for severe neuromuscular and neurological diseases.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Rampart Bioscience specializes in the development of gene medicines aimed at providing long-lasting treatments for a broad array of diseases. The company has created proprietary development and delivery platforms that enable the production of optimized therapeutics in a non-viral format. By integrating expertise from various scientific disciplines, such as gene delivery, protein sciences, and clinical translation, Rampart Bioscience focuses on assisting patients with genetically driven conditions, ultimately enhancing the effectiveness and safety of therapeutic interventions.

ExcepGen Ltd is a biotechnology company focused on developing full-length human proteins for drug discovery. Founded in 2017 and based in London, the company employs engineered human cell lines to produce these proteins, facilitating research and development in therapeutics. By utilizing proprietary technology, ExcepGen aims to enhance the effectiveness of mRNA therapeutics, optimizing cellular responses to improve therapeutic outcomes. This approach allows laboratories to minimize development time, accelerate high-throughput screening processes, and make informed decisions regarding their therapeutic pipelines. With a commitment to addressing fundamental challenges in drug discovery, ExcepGen is positioned to contribute significantly to the advancement of innovative treatments.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, established in 2019, focused on developing adeno-associated virus (AAV)-based gene therapies for monogenic diseases affecting the central nervous system (CNS). The company’s product pipeline includes several therapies currently in development, such as TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha Gene Therapies aims to translate innovative treatments from research to clinical practice efficiently, leveraging a strategic partnership with The University of Texas Southwestern Medical Center to enhance its development and commercialization capabilities. The company's mission is to create curative medicines that target both rare and prevalent CNS conditions, thereby addressing severe and life-threatening diseases.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

Jnana Therapeutics Inc. is a biotechnology company based in Boston, Massachusetts, that focuses on drug discovery through its innovative platform targeting solute carrier (SLC) transporters. Established in 2016, the company aims to improve patient outcomes by developing small molecule therapies for various diseases, including those related to immunometabolism, lysosomal function, and mucosal defense. By utilizing advanced chemoproteomics techniques, Jnana accelerates the identification of therapeutic targets, addressing significant unmet medical needs in areas such as immuno-oncology, inflammatory disorders, and neurological diseases. The company's approach seeks to enhance drug discovery efficiency by comprehensively targeting the vast family of membrane proteins associated with SLC transporters.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

NAPIGEN is a biotechnology company focused on innovative genome engineering across a variety of organisms, including plants, microbes, and animals. The company specializes in targeting organelles such as mitochondria and chloroplasts, which contain DNA essential for cellular energy production. By harnessing the capabilities of these organelles, NAPIGEN aims to enhance biological functions and contribute to advancements in agriculture, environmental sustainability, and other sectors, ultimately improving the quality of life in society.

Wave Life Sciences Ltd. is a clinical-stage genetic medicine company focused on developing innovative RNA-based therapies. Utilizing its proprietary PRISM platform, the company designs, optimizes, and produces stereopure oligonucleotides aimed at addressing genetic defects. By targeting ribonucleic acid (RNA), Wave aims to either diminish the production of harmful proteins or convert dysfunctional proteins into functional ones. The company's primary focus is on neurology, particularly disorders affecting the central and neuromuscular systems. Wave Life Sciences has established research and collaboration agreements with major pharmaceutical companies, including Pfizer and Takeda, to further advance its therapeutic programs. Founded in 2012 and headquartered in Singapore, Wave Life Sciences is dedicated to delivering transformative treatments for patients with genetically defined diseases.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

Ansa Biotechnologies, Inc. is a biotechnology company based in Berkeley, California, focused on developing innovative DNA synthesis technology utilizing enzymes. Established in 2018, the company aims to improve upon traditional chemical methods of DNA synthesis that have remained largely unchanged for decades. Ansa's enzymatic approach offers significant advantages, including increased speed, accuracy, and cleanliness in the production of DNA fragments such as oligonucleotides. This technology enhances the capabilities of biological research, therapeutics, diagnostics, and biomanufacturing by enabling the synthesis of gene-length oligonucleotides. Through its advancements, Ansa Biotechnologies seeks to facilitate innovation in the fields of biological research and engineering, ultimately providing valuable services to medical researchers and contributing to the broader biotechnology landscape.

Synthego Corporation is a genome engineering company focused on advancing life science research and therapeutic development to improve human health. Founded in 2012 and headquartered in Redwood City, California, Synthego offers a comprehensive platform that integrates proprietary hardware, software, bioinformatics, chemistries, and molecular biology. The company's product portfolio includes engineered cells, CRISPR kits, and bioinformatics tools designed for various applications such as pathway analysis, target validation, disease modeling, and diagnostics. By leveraging machine learning and automation, Synthego enables researchers in biotechnology and academia to conduct efficient and precise gene editing, facilitating rapid advancements in drug discovery and development. The company serves a global customer base, distributing its products through various channels, including online sales and partnerships in multiple countries.

Metagenomi, Inc. is a biotechnology company focused on developing gene editing systems aimed at treating genetic diseases. Founded in 2016 and based in Emeryville, California, Metagenomi utilizes metagenomics and machine learning to uncover novel genome editing technologies derived from previously uncharacterized organisms. The company's innovative discovery platform enables the creation of highly efficient and specific gene editing tools, including programmable nucleases, base editors, and advanced integration systems such as prime editing and CRISPR-associated transposases. By harnessing these naturally evolved systems, Metagenomi seeks to address a wide range of genetic mutations that traditional genome engineering approaches have struggled to target, with the ultimate goal of providing transformative therapies for patients globally.

Jnana Therapeutics Inc. is a biotechnology company based in Boston, Massachusetts, that focuses on drug discovery through its innovative platform targeting solute carrier (SLC) transporters. Established in 2016, the company aims to improve patient outcomes by developing small molecule therapies for various diseases, including those related to immunometabolism, lysosomal function, and mucosal defense. By utilizing advanced chemoproteomics techniques, Jnana accelerates the identification of therapeutic targets, addressing significant unmet medical needs in areas such as immuno-oncology, inflammatory disorders, and neurological diseases. The company's approach seeks to enhance drug discovery efficiency by comprehensively targeting the vast family of membrane proteins associated with SLC transporters.

GentiBio, Inc. is a biotherapeutics company focused on developing engineered regulatory T cells (EngTregs) aimed at treating autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and headquartered in Boston, Massachusetts, with additional locations in Israel and Seattle, GentiBio utilizes a proprietary platform that combines autologous and allogeneic EngTregs technologies. This innovative approach is designed to restore immune tolerance and address limitations present in current regulatory T-cell therapies. By targeting the underlying causes of diseases related to immune system dysfunction, GentiBio aims to provide effective therapeutic solutions for conditions that arise from immune system overshooting or malfunctioning. The company was co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of this emerging field.

PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing nucleic acid therapeutics, particularly antisense oligonucleotides. Founded in 2018, the company aims to enhance the delivery and efficacy of these therapies through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative platform utilizes engineered peptides designed to improve tissue penetration, cellular uptake, and nuclear delivery of oligonucleotides. PepGen's technology has shown promise in preclinical studies, demonstrating the ability to transport therapeutics effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. The company's mission is to unlock the full potential of nucleic acid therapeutics to transform the treatment landscape for severe neuromuscular and neurological diseases.

Element Biosciences, Inc., established in 2017 and headquartered in San Diego, California, specializes in developing innovative genetic analysis tools for research and diagnostic markets. The company's core business revolves around its disruptive DNA sequencing technology, which encompasses surface chemistry, sequencing chemistry, detection methods, and data analysis. Element Biosciences aims to enhance accessibility to next-generation sequencing by offering a modular, high-performing platform that delivers high-quality data and workflow flexibility, thereby reducing run and capital costs.

Affinia Therapeutics Inc. is focused on developing gene therapies for various diseases, particularly those affecting the muscles and central nervous system. The company specializes in creating adeno-associated virus (AAV) vectors, leveraging advanced techniques in synthetic and systems biology, as well as high-throughput screening and tissue and single-cell resolution. Founded in 2019 and based in Waltham, Massachusetts, Affinia aims to address significant unmet medical needs by providing rationally designed gene therapies with improved properties. The company's innovative approach seeks to offer effective treatments for devastating conditions that currently lack adequate therapeutic options.

Scribe Therapeutics Inc. is a biotechnology company focused on developing innovative therapeutics based on CRISPR technology to address genetic disorders. Founded in 2017 and headquartered in Berkeley, California, the company utilizes its proprietary X-Editing (XE) platform, which features highly engineered CRISPR enzymes designed to improve the efficacy, specificity, and deliverability of genome editing. Scribe Therapeutics aims to overcome existing limitations in genetic medicine by creating custom-engineered enzymes and advanced delivery systems. Their approach seeks to establish CRISPR-based therapies as a new standard in clinical care, potentially transforming the treatment landscape for various conditions, including immuno-oncology, degenerative disorders, and enzyme replacement therapy. By prioritizing therapeutic precision and allele-specific targeting, Scribe Therapeutics is committed to expanding access to groundbreaking treatments that can significantly impact patient lives.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that specializes in targeted DNA integration to develop innovative therapies for severe diseases. The company utilizes a precision gene editing approach to precisely correct genetic mutations, replace malfunctioning genes, and insert therapeutic genetic sequences at specific locations within the genome. Its technology aims to transform treatment paradigms by enabling the targeted integration of genetic payloads, which could potentially cure a range of serious and life-threatening conditions. Graphite Bio was formerly known as Integral Medicines, Inc. before rebranding in August 2020, and it was incorporated in 2019.

Century Therapeutics, Inc. is a biotechnology company focused on developing innovative cell therapies derived from induced pluripotent stem cells (iPSCs) to treat hematologic and solid malignancies. Founded in 2018 and headquartered in Philadelphia, Pennsylvania, the company utilizes a specialized platform that integrates gene editing and protein engineering to create allogeneic therapies. This platform allows for the generation of master cell banks of modified immune effector cells, specifically engineered to optimize performance and reduce the risk of rejection by the host immune system. By employing advanced technologies such as CRISPR-mediated gene editing and proprietary chimeric antigen receptors, Century Therapeutics aims to address significant unmet medical needs in cancer and other diseases.

DTx Pharma, LLC is a biotechnology company focused on developing and commercializing fatty acid-conjugated peptide therapeutics for patients with rare and chronic diseases. The company specializes in RNA-based therapeutics, utilizing a delivery technology platform that enhances the distribution of nucleic acid drugs to various tissues and organ systems beyond the liver. This innovative approach addresses limitations associated with previous-generation RNA delivery methods, such as poor pharmacokinetics and inadequate cellular uptake. DTx Pharma's products target conditions including retinitis pigmentosa, Duchenne muscular dystrophy, and central nervous system disorders. Founded in 2017 and based in San Diego, California, DTx Pharma aims to provide personalized treatment options across multiple therapeutic areas.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

Design Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing innovative therapies for degenerative disorders linked to nucleotide repeat expansions. Founded in 2017 and headquartered in Solana Beach, California, the company is advancing its lead program aimed at treating Friedreich’s ataxia. Additionally, Design Therapeutics is exploring treatments for other conditions such as Fragile X syndrome and myotonic dystrophy, utilizing its proprietary GeneTACTM technology, which involves small-molecule gene-targeted therapeutics designed to modify the underlying causes of these inherited diseases. The company is committed to addressing serious monogenic disorders driven by nucleotide repeat mutations through its ongoing research and development efforts.

Verve Therapeutics, Inc. is a genetic medicines company focused on innovative treatments for cardiovascular disease through gene editing. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company aims to shift the management of cardiovascular conditions from chronic treatments to single-course gene editing therapies. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are known to significantly impact blood lipid levels, a major factor in cardiovascular disease. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance its gene editing delivery technologies. The company is staffed by a team of experts in cardiovascular medicine, human genetics, and drug development, positioning itself to address the challenges of cardiovascular health effectively.

Locanabio, Inc. is a biotechnology company based in San Diego, California, founded in 2016. It specializes in developing RNA-targeted gene therapies aimed at treating a range of underserved diseases, particularly in the areas of neuromuscular, neurodegenerative, and retinal conditions. The company's innovative platform focuses on modifying disease-causing RNA, allowing for the identification and correction of specific RNA sequences associated with rare genetic disorders. This approach distinguishes Locanabio's therapies from traditional DNA-targeted methods, offering a unique solution for patients with severe and challenging health issues. The company, which rebranded from Locana, Inc. in July 2020, is dedicated to advancing therapeutic candidates that address significant medical needs.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing nucleic acid therapeutics, particularly antisense oligonucleotides. Founded in 2018, the company aims to enhance the delivery and efficacy of these therapies through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative platform utilizes engineered peptides designed to improve tissue penetration, cellular uptake, and nuclear delivery of oligonucleotides. PepGen's technology has shown promise in preclinical studies, demonstrating the ability to transport therapeutics effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. The company's mission is to unlock the full potential of nucleic acid therapeutics to transform the treatment landscape for severe neuromuscular and neurological diseases.

Metagenomi, Inc. is a biotechnology company focused on developing gene editing systems aimed at treating genetic diseases. Founded in 2016 and based in Emeryville, California, Metagenomi utilizes metagenomics and machine learning to uncover novel genome editing technologies derived from previously uncharacterized organisms. The company's innovative discovery platform enables the creation of highly efficient and specific gene editing tools, including programmable nucleases, base editors, and advanced integration systems such as prime editing and CRISPR-associated transposases. By harnessing these naturally evolved systems, Metagenomi seeks to address a wide range of genetic mutations that traditional genome engineering approaches have struggled to target, with the ultimate goal of providing transformative therapies for patients globally.

AavantiBio, Inc. is a biopharmaceutical company based in Gainesville, Florida, focused on developing gene transfer and gene editing therapies for rare genetic diseases. Established in 2019, the company primarily targets Friedreich’s Ataxia, a severe inherited condition that leads to significant cardiac and central nervous system dysfunction. In addition to addressing this specific disorder, AavantiBio is advancing a pipeline of innovative gene therapies aimed at treating various autoimmune diseases and other conditions with unmet medical needs. Through its commitment to research and development, the company seeks to improve the lives of patients affected by these debilitating genetic disorders.

Synthego Corporation is a genome engineering company focused on advancing life science research and therapeutic development to improve human health. Founded in 2012 and headquartered in Redwood City, California, Synthego offers a comprehensive platform that integrates proprietary hardware, software, bioinformatics, chemistries, and molecular biology. The company's product portfolio includes engineered cells, CRISPR kits, and bioinformatics tools designed for various applications such as pathway analysis, target validation, disease modeling, and diagnostics. By leveraging machine learning and automation, Synthego enables researchers in biotechnology and academia to conduct efficient and precise gene editing, facilitating rapid advancements in drug discovery and development. The company serves a global customer base, distributing its products through various channels, including online sales and partnerships in multiple countries.

GentiBio, Inc. is a biotherapeutics company focused on developing engineered regulatory T cells (EngTregs) aimed at treating autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and headquartered in Boston, Massachusetts, with additional locations in Israel and Seattle, GentiBio utilizes a proprietary platform that combines autologous and allogeneic EngTregs technologies. This innovative approach is designed to restore immune tolerance and address limitations present in current regulatory T-cell therapies. By targeting the underlying causes of diseases related to immune system dysfunction, GentiBio aims to provide effective therapeutic solutions for conditions that arise from immune system overshooting or malfunctioning. The company was co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of this emerging field.

Element Biosciences, Inc., established in 2017 and headquartered in San Diego, California, specializes in developing innovative genetic analysis tools for research and diagnostic markets. The company's core business revolves around its disruptive DNA sequencing technology, which encompasses surface chemistry, sequencing chemistry, detection methods, and data analysis. Element Biosciences aims to enhance accessibility to next-generation sequencing by offering a modular, high-performing platform that delivers high-quality data and workflow flexibility, thereby reducing run and capital costs.

Akouos, Inc. is a biotechnology company based in Boston, Massachusetts, dedicated to developing gene therapies aimed at restoring, improving, and preserving hearing for individuals affected by various inner ear disorders. Founded in 2016, the company utilizes an innovative platform that includes a proprietary adeno-associated viral vector library and a unique delivery method. Its leading product candidate, AK-OTOF, targets hearing loss resulting from mutations in the OTOF gene. Akouos focuses on genetically-defined patient populations, addressing conditions ranging from single genetic mutations to hearing loss caused by ototoxic drugs and aging. The company has established strategic partnerships with key institutions, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing precision genetic therapies for sensorineural hearing loss.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Prevail Therapeutics Inc. is a biotechnology company headquartered in New York, specializing in the development and commercialization of gene therapies targeting neurodegenerative diseases. Founded in 2017, the company focuses on adeno-associated virus (AAV)-based therapies, with its lead candidate, PR001, currently undergoing Phase I/II clinical trials for Parkinson's disease associated with GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is advancing PR006 for frontotemporal dementia linked to GRN mutations and PR004 for various synucleinopathies. The company's mission is to provide disease-modifying treatments for patients suffering from these challenging conditions.

Stoke Therapeutics, Inc. is an early-stage biopharmaceutical company headquartered in Bedford, Massachusetts, focused on developing novel antisense oligonucleotide medicines to address the underlying causes of severe genetic diseases. The company employs its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) technology to enhance gene expression by targeting RNA splicing, thereby selectively restoring protein levels. Its lead product candidate, STK-001, is currently in clinical testing for the treatment of Dravet syndrome, a serious and progressive genetic epilepsy. Established in 2014, Stoke Therapeutics also collaborates with Invitae Corporation to provide genetic testing services, further supporting its mission to advance gene-targeted therapies for patients with severe genetic conditions.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

Akouos, Inc. is a biotechnology company based in Boston, Massachusetts, dedicated to developing gene therapies aimed at restoring, improving, and preserving hearing for individuals affected by various inner ear disorders. Founded in 2016, the company utilizes an innovative platform that includes a proprietary adeno-associated viral vector library and a unique delivery method. Its leading product candidate, AK-OTOF, targets hearing loss resulting from mutations in the OTOF gene. Akouos focuses on genetically-defined patient populations, addressing conditions ranging from single genetic mutations to hearing loss caused by ototoxic drugs and aging. The company has established strategic partnerships with key institutions, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing precision genetic therapies for sensorineural hearing loss.

Precision BioSciences, Inc. is a biotechnology company focused on genome editing, headquartered in Durham, North Carolina. The company utilizes its proprietary ARCUS technology to develop therapeutic products aimed at treating human diseases and improving food and agricultural solutions. Precision BioSciences operates through two segments: Therapeutic and Food. The Therapeutic segment is particularly dedicated to advancing allogeneic CAR T immunotherapies for cancer treatment, including candidates like PBCAR0191 and PBCAR20A, which target specific tumor antigens. The company is also involved in in vivo gene correction and is exploring treatments for conditions such as acute lymphoblastic leukemia and chronic lymphocytic leukemia. Additionally, the Food segment develops innovative nutrition products. Precision BioSciences has formed strategic collaborations, including agreements with Shire Plc and Gilead Sciences, to enhance its research and development capabilities. Founded in 2006, the company aims to harness its next-generation gene editing technology to address pressing health challenges.

Prevail Therapeutics Inc. is a biotechnology company headquartered in New York, specializing in the development and commercialization of gene therapies targeting neurodegenerative diseases. Founded in 2017, the company focuses on adeno-associated virus (AAV)-based therapies, with its lead candidate, PR001, currently undergoing Phase I/II clinical trials for Parkinson's disease associated with GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is advancing PR006 for frontotemporal dementia linked to GRN mutations and PR004 for various synucleinopathies. The company's mission is to provide disease-modifying treatments for patients suffering from these challenging conditions.

Dyne Therapeutics is a biotechnology company based in Waltham, Massachusetts, dedicated to developing targeted therapies for genetically driven muscle diseases. Founded in 2017, the company focuses on advancing treatments for conditions such as myotonic dystrophy type 1, Duchenne muscular dystrophy, and facioscapulohumeral dystrophy, among others. Utilizing its innovative FORCE platform, Dyne Therapeutics aims to deliver disease-modifying therapies with enhanced precision to skeletal, cardiac, and smooth muscle tissues. The company's lead product candidates, DYNE-101 and DYNE-251, are currently undergoing phase 1/2 clinical trials, reflecting its commitment to addressing the unmet medical needs of patients with serious muscle disorders.

Dicerna Pharmaceuticals is a biotechnology company dedicated to the discovery and development of innovative treatments for rare inherited diseases and other conditions affecting the liver. Utilizing its proprietary ribonucleic acid interference (RNAi) technology, specifically the GalXC platform, Dicerna focuses on silencing disease-causing genes to develop pharmaceuticals for various therapeutic areas, including liver diseases, cardiovascular diseases, and cancers. Key product candidates include nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B virus infection, and additional programs targeting undisclosed rare liver diseases. The company has established strategic collaborations with several major pharmaceutical firms to enhance its research and development efforts. Founded in 2006 and headquartered in Lexington, Massachusetts, Dicerna Pharmaceuticals aims to address significant unmet medical needs through its innovative approaches.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Audentes Therapeutics, a clinical stage biotechnology company based in San Francisco, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

AveXis, now known as Novartis Gene Therapies, is a biotechnology company focused on developing and commercializing gene therapies aimed at treating rare and life-threatening neurological genetic disorders. Acquired by Novartis in 2018, the company specializes in motor neuron cell-targeted gene replacement therapies for conditions such as spinal muscular atrophy, Rett syndrome, and amyotrophic lateral sclerosis. By addressing these severe diseases, which often result in progressive muscle weakness, paralysis, and death, AveXis plays a crucial role in enhancing treatment options for affected patients and their families.

Wave Life Sciences Ltd. is a clinical-stage genetic medicine company focused on developing innovative RNA-based therapies. Utilizing its proprietary PRISM platform, the company designs, optimizes, and produces stereopure oligonucleotides aimed at addressing genetic defects. By targeting ribonucleic acid (RNA), Wave aims to either diminish the production of harmful proteins or convert dysfunctional proteins into functional ones. The company's primary focus is on neurology, particularly disorders affecting the central and neuromuscular systems. Wave Life Sciences has established research and collaboration agreements with major pharmaceutical companies, including Pfizer and Takeda, to further advance its therapeutic programs. Founded in 2012 and headquartered in Singapore, Wave Life Sciences is dedicated to delivering transformative treatments for patients with genetically defined diseases.

ARCA biopharma, Inc. is a Colorado-based biopharmaceutical company developing genetically-targeted therapies for cardiovascular diseases. ARCA’s principal focus is to develop personalized therapies for the treatment of cardiovascular disease through the use of genetics. The Company’s business focus combines expertise in cardiovascular pathophysiology, molecular genetics and clinical development.

Calimmune, Inc. is a clinical-stage company based in Tucson, Arizona, specializing in gene therapies for individuals living with human immunodeficiency virus (HIV). The company is developing Cal-1, a gene-based stem cell therapy designed to protect patients from progressing to AIDS. This innovative therapy aims to control HIV infection through a dual mechanism: it reduces the production of CCR5, a protein that facilitates HIV's ability to infect cells, and prevents viral fusion, thereby blocking the virus from entering cells. Currently, Cal-1 is undergoing Phase I/II clinical studies to evaluate its effectiveness. Calimmune focuses on addressing the diverse needs of individuals at various stages of HIV infection, emphasizing the importance of tailored treatments for those with different levels of treatment experience. Founded in 2000, Calimmune operates as a subsidiary of CSL Behring LLC.

Precision BioSciences, Inc. is a biotechnology company focused on genome editing, headquartered in Durham, North Carolina. The company utilizes its proprietary ARCUS technology to develop therapeutic products aimed at treating human diseases and improving food and agricultural solutions. Precision BioSciences operates through two segments: Therapeutic and Food. The Therapeutic segment is particularly dedicated to advancing allogeneic CAR T immunotherapies for cancer treatment, including candidates like PBCAR0191 and PBCAR20A, which target specific tumor antigens. The company is also involved in in vivo gene correction and is exploring treatments for conditions such as acute lymphoblastic leukemia and chronic lymphocytic leukemia. Additionally, the Food segment develops innovative nutrition products. Precision BioSciences has formed strategic collaborations, including agreements with Shire Plc and Gilead Sciences, to enhance its research and development capabilities. Founded in 2006, the company aims to harness its next-generation gene editing technology to address pressing health challenges.

Dimension Therapeutics, Inc. is a gene therapy company dedicated to developing innovative treatments for rare and metabolic diseases linked to liver function. Founded in 2013 and based in Cambridge, Massachusetts, the company has established a robust pipeline of gene therapy candidates. Its lead products include DTX101 for hemophilia B and DTX201 for hemophilia A, alongside other programs targeting conditions such as ornithine transcarbamylase deficiency, glycogen storage disease type Ia, phenylketonuria, Wilson disease, and citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, leveraging its extensive patent portfolio related to NAV vector technology. This partnership enhances Dimension's ability to advance its gene therapy initiatives, particularly in the realm of adeno-associated virus therapeutics. In 2017, Dimension Therapeutics became a subsidiary of Ultragenyx Pharmaceutical Inc.

Natera, Inc. is a diagnostic and research company specializing in genetic testing services, particularly in the areas of preconception and prenatal diagnostics. Founded in 2003 and headquartered in San Carlos, California, Natera offers a range of products including the Panorama non-invasive prenatal test, which screens for chromosomal abnormalities in fetuses, and Vistara, a test for single-gene mutations. Additionally, the company provides Horizon carrier screening to assess carrier status for various genetic diseases, and Spectrum, which analyzes genetic conditions during in vitro fertilization. Natera's Anora product analyzes fetal chromosomes to understand miscarriage causes, while its non-invasive paternity tests determine parentage through fetal DNA analysis. The company's Signatera technology focuses on circulating tumor DNA to monitor cancer recurrence. Natera distributes its products through a direct sales force and a network of approximately 100 laboratory and distribution partners, both domestically and internationally. The company collaborates with BGI Genomics to develop genetic testing assays and has partnered with Foundation Medicine for personalized cancer monitoring assays.

Wave Life Sciences Ltd. is a clinical-stage genetic medicine company focused on developing innovative RNA-based therapies. Utilizing its proprietary PRISM platform, the company designs, optimizes, and produces stereopure oligonucleotides aimed at addressing genetic defects. By targeting ribonucleic acid (RNA), Wave aims to either diminish the production of harmful proteins or convert dysfunctional proteins into functional ones. The company's primary focus is on neurology, particularly disorders affecting the central and neuromuscular systems. Wave Life Sciences has established research and collaboration agreements with major pharmaceutical companies, including Pfizer and Takeda, to further advance its therapeutic programs. Founded in 2012 and headquartered in Singapore, Wave Life Sciences is dedicated to delivering transformative treatments for patients with genetically defined diseases.

Moderna, Inc. is a biotechnology company established in 2010 and headquartered in Cambridge, Massachusetts. The company specializes in developing therapeutics and vaccines using messenger RNA (mRNA) technology, which provides instructions for cells to produce proteins essential for various biological functions. Moderna has a diverse pipeline, with numerous programs targeting infectious diseases, immuno-oncology, rare diseases, and cardiovascular conditions. As of September 2024, it has 40 mRNA development candidates in clinical trials across multiple therapeutic areas. The company's mRNA technology gained significant recognition with the authorization of its COVID-19 vaccine in December 2020. Moderna maintains strategic alliances with various organizations, including major pharmaceutical companies and research institutions, to enhance its research and development efforts.

Arrowhead Pharmaceuticals, Inc. is a biotechnology company based in Pasadena, California, focused on developing innovative medicines for the treatment of intractable diseases, particularly those with a genetic origin characterized by the overproduction of specific proteins. The company's diverse pipeline includes RNA interference (RNAi) therapeutics such as ARO-AAT for liver diseases associated with alpha-1 antitrypsin deficiency, ARO-APOC3 for hypertriglyceridemia, and ARO-HIF2 for clear cell renal cell carcinoma, among others. Arrowhead is also exploring treatments for various conditions, including chronic hepatitis B virus infection and cardiovascular diseases. The company collaborates with prominent pharmaceutical firms, including Janssen Pharmaceuticals and Takeda Pharmaceuticals, to develop additional RNAi therapeutic candidates. Originally founded as Arrowhead Research Corporation in 1989, it adopted its current name in April 2016.

Dicerna Pharmaceuticals is a biotechnology company dedicated to the discovery and development of innovative treatments for rare inherited diseases and other conditions affecting the liver. Utilizing its proprietary ribonucleic acid interference (RNAi) technology, specifically the GalXC platform, Dicerna focuses on silencing disease-causing genes to develop pharmaceuticals for various therapeutic areas, including liver diseases, cardiovascular diseases, and cancers. Key product candidates include nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B virus infection, and additional programs targeting undisclosed rare liver diseases. The company has established strategic collaborations with several major pharmaceutical firms to enhance its research and development efforts. Founded in 2006 and headquartered in Lexington, Massachusetts, Dicerna Pharmaceuticals aims to address significant unmet medical needs through its innovative approaches.

Bluebird bio, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on researching, developing, and commercializing gene therapies for severe genetic diseases and cancer. The company's gene therapy programs include LentiGlobin, aimed at treating β-thalassemia and sickle cell disease, and Lenti-D for cerebral adrenoleukodystrophy. In oncology, bluebird bio is developing chimeric antigen receptor T (CAR T) cell therapies, specifically bb2121 and bb21217, for multiple myeloma. The company has formed strategic collaborations with several organizations, including Bristol-Myers Squibb and Regeneron Pharmaceuticals, to advance gene therapies in oncology and immune cell therapies. Additionally, bluebird bio collaborates with various partners to explore T cell receptor product candidates and in vivo genome editing treatments for genetic disorders. Established in 1992 and originally known as Genetix Pharmaceuticals, the company transitioned to its current name in 2010.

CellScape Corporation (formerly Saryna Medical Corp) is a privately funded company headquartered in Newark, California, in the heart of the biotech corridor of the San Francisco Bay Area. The Company has developed whole fetal cell technology to transform prenatal genetic testing using a simple blood test. Their vision is to make comprehensive prenatal genetic testing available to every woman without requiring an invasive procedure that might put the fetus at risk. Their leadership team is accomplished in bringing innovative, cost-effective medical and molecular diagnostics to market.